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1.
Acta Pharmaceutica Sinica ; (12): 574-580, 2024.
Article in Chinese | WPRIM | ID: wpr-1016626

ABSTRACT

Parkinson's disease (PD) is a chronic neurodegenerative disease. At present, levodopa and other drugs are mainly used for dopamine supplementation therapy. However, the absorption of levodopa in the gastrointestinal tract is unstable and its half-life is short, and long-term use of levodopa will lead to the end-of-dose deterioration, dyskinesia, the "ON-OFF" phenomenon and other symptoms. Therefore, new preparations need to be developed to improve drug efficacy, reduce side effects or improve compliance of patients. Based on the above clinical needs, this review briefly introduced the preparation modification strategies for the treatment of PD through case analysis, in order to provide references for the research and development of related preparations.

2.
Chinese Pharmacological Bulletin ; (12): 234-242, 2024.
Article in Chinese | WPRIM | ID: wpr-1013621

ABSTRACT

Aim To investigate the regulatory effect of Cortaetin on pathological myocardial hypertrophy induced by isoprenaline (ISO) and the underlying mechanism. Methods ISO was used to stimulate neonatal rat cardiomyocytes for 24 h, and myocardial hypertrophy model was established at the cellular level. C57BL/6 mice were injected subcutaneously with ISO for one week to establish myocardial hypertrophy model at animal level. RT-qPCR was used to detect the changes of mRNA and Western blot was used to detect the changes of relative protein content. Immunofluorescence was used to measure the subcellular location of Cortaetin and the change of its expression. The overex-pression of Cortaetin by adenovirus infection and the knockdown of Cortaetin by transfection of small interfering RNA were studied. Results On the cellular and animal levels, ISO-induced myocardial hypertrophy models were successfully established, and it was observed that ISO caused the decrease of Cortaetin and N-cadherin protein levels. Overexpression of Cortaetin could reverse the decrease of N-cadherin protein level and myocardial hypertrophy caused by ISO. Knockdown of Cortaetin showed the opposite effect. Conclusion Cortaetin, in combination with N-cadherin, may play a role in combating myocardial hypertrophy by enhancing the connections between cardiomyocytes.

3.
Chinese Journal of Pathology ; (12): 64-70, 2024.
Article in Chinese | WPRIM | ID: wpr-1012426

ABSTRACT

Objective: To investigate the clinicopathological features, immunophenotype, diagnosis and differential diagnosis of SRF-rearranged cellular perivascular myoid tumor. Methods: Two cases of SRF-rearranged cellular perivascular myoid tumor diagnosed in the Department of Pathology, Fudan University Shanghai Cancer Center from October 2021 to March 2022 were collected. Immunohistochemical staining, fluorescence in-situ hybridization (FISH) and next-generation sequencing (NGS) were performed, and the literature was reviewed. Results: Case 1, a 3-month-old boy presented with a painless tumor of the scalp, measuring about 2 cm in diameter. Case 2, a 3-year-old girl complained with a painless tumor of the knee, measuring approximately 1.5 cm in diameter. Microscopically, the tumor had a clear boundary and showed multinodular growth. The tumor was mainly composed of spindle cells arranged in long intersecting fascicles associated with thin, slit-like or branching ectatic vessels, focally forming hemangiopericytoma-like appearance. The tumor cells were abundant, but there was no obvious atypia. Mitotic figures (3-4/10 HPF) were noted. H-caldesmon and SMA were positive in both cases. Case 1 showed diffuse and strong positivity for Desmin, and focally for CKpan. Ki-67 proliferation index was 20% and 30%, respectively. FISH displayed NCOA2 gene translocation in case 1 and the RELA gene translocation in case 2. NGS detected the SRF-NCOA2 gene fusion in case 1 and the SRF-RELA gene fusion in case 2. Both patients underwent local excisions. During the follow-up of 5-14 months, case 1 had no local recurrence, while case 2 developed local recurrence 1 year post operatively. Conclusions: SRF-rearranged cellular perivascular myoid tumor is a novel variant of perivascular cell tumor, which tends to occur in children and adolescents. The tumor forms a broad morphologic spectrum ranging from a pericytic pattern to a myoid pattern, and include hybrid tumors with a mixture of pericytic and myoid patterns. Due to its diffuse hypercellularity and increased mitotic figures and smooth muscle-like immunophenotype, the tumor is easy to be misdiagnosed as myogenic sarcomas. The tumor usually pursues a benign clinical course and rare cases may locally recur.


Subject(s)
Child, Preschool , Female , Humans , Infant , Male , Biomarkers, Tumor/analysis , Calmodulin-Binding Proteins , China , Hemangiopericytoma/pathology , Sarcoma/pathology , Soft Tissue Neoplasms/pathology
4.
Clinical Medicine of China ; (12): 53-56, 2023.
Article in Chinese | WPRIM | ID: wpr-992464

ABSTRACT

Objective:To investigate the clinical and genetic characteristics of Prader-Willi syndrome (PWS).Methods:The clinical data and genetic characteristics of 2 children with PWS diagnosed in Hebei Provincial People's Hospital were retrospectively analyzed, and the relevant literature was reviewed.Results:Case 1, male, aged 6 years and 3 months, was presented to the hospital because of short stature, mild mental retardation, dysarthria, scoliosis, cryptorchidism, micropenis, long skull, narrow face, almond eyes, small mouth, thin upper lip, downward corners of the mouth, fair skin. He had hypotonia and feeding difficulties in infancy, and gradually became hyperappetitive. Bilateral cryptorchidism surgery was performed at 1.5 years old, but the effect was not good. Case 2, male, aged 4 years, presented to the hospital mainly due to obesity, hyperappetite, excessive weight gain, backward language and cognitive function, dysarthria, and scoliosis.The infant had feeding difficulties in the early stage, and bilateral cryptorchidism surgery at the age of 2 was not effective.Methylation specific polymerase chain reaction and methylation specific multilink probe amplification were used to detect the loss of the parent fragment in the key region (15q11-13) of PWS, which confirmed Prader-Willi syndrome.Conclusion:PWS is a rare hereditary disease with complex and diverse clinical manifestations and different characteristics in different age groups. It is highly susceptible to unexplained hypotonia and feeding difficulties in infancy. Children with short stature and obesity should be alert to the disease, which can be clearly diagnosed by molecular genetic techniques.

5.
Chinese Pediatric Emergency Medicine ; (12): 122-125, 2023.
Article in Chinese | WPRIM | ID: wpr-990489

ABSTRACT

Objective:To investigate the risk factors of recurrence of febrile seizures within 24 hours, so as to provide clinical evidence for early identification of children with risk factors and taking interventions.Methods:A total of 384 children with febrile seizures admitted to the Department of Pediatrics at Hebei General Hospital from June 2019 to June 2021 were selected as the study subjects, and were divided into single seizure group and recurrent seizures group.The clinical data of two groups and the risk factors of recurrent seizures were analyzed retrospectively.Results:A total of 384 children, aging from six months to five years, were diagnosed with febrile seizures.There were 296 cases in the single seizure group and 88 cases in the recurrent seizures group.First seizure, the age of the first sezures, temperature, duration of seizure ≥15 minutes, positive family history and C-reactive protein levels showed statistically significant differences between two groups(all P<0.05). Logistic regression analysis showed that non-first seizure( OR=2.085, 95% CI 1.232-3.529, P=0.006), the age of first seizure( OR=0.970, 95% CI 0.948-0.993, P=0.010), duration of seizure ≥15 minutes( OR=3.587, 95% CI 1.497-8.596, P=0.004) and positive family history( OR=1.892, 95% CI 1.126-3.180, P=0.016) were risk factors of recurrence of febrile seizures within 24 hours.The ROC curve analysis showed that the combination of four risk factors had a higher predictive value, and the area under curve was 0.974. Conclusion:Non-first seizure, the age of first seizure, cluration of seizure ≥15 minutes and positive family history are the risk factors of recurrence of febrile seizures within 24 hours.Children with four risk factors are more likely to have recurrent seizure, and could be used as an indicator for individualized prediction.

6.
Chinese Journal of Applied Clinical Pediatrics ; (24): 54-59, 2023.
Article in Chinese | WPRIM | ID: wpr-989990

ABSTRACT

Objective:To investigate the incidence rate and gene variation of methylmalonic academia (MMA) in Ji′nan city by analyzing biochemical and genetic screening results, and to explore the carrier frequency of MMA-related pathogenic genes in the population in Ji′nan.Methods:The children diagnosed with MMA by tandem mass spectrometry screening in Ji′nan Neonatal Disease Screening Centre from May 2011 to May 2022 were enrolled in this study.Their genetic test results were retrospectively analyzed and summarized.The dried heel blood tablets collected from 6 800 newborns were tested for neonatal gene screening. MMAA, MMAB, MMACHC and MMUT genes in 4 800 cases were detected by high-throughput sequencing+ target area capture technology.Ultra-multiplex polymerase chain reaction+ target gene locus capture technology was used to detect 174 target loci of 8 genes related to MMA in 2 000 cases.The hotspot mutation and related gene carrier rate of MMA were analyzed. Results:A total of 367 452 newborns were screened by tandem mass spectrometry, and 103 cases (56 males and 47 females) were diagnosed with MMA by screening.The estimated incidence of MMA was 1∶3 567.Among the 103 MMA cases, 76 were genetically diagnosed, and 4 gene variants of MMA ( MMAHC, MMUT, MMAA, MMADHC) were identified.A total of 6 800 neonates underwent neonatal genetic screening.Three of them were diagnosed with MMA.About 318 infants carried pathogenic variants of MMA, with a total carrier rate of 4.68%.Specifically, the carrier rates of MMACHC and MMUT gene variants were 3.09%(210/6 800) and 1.43% (97/6 800), respectively. Conclusions:MMA is the most common organic acid metabolism disorder in our country.The incidence and carrier rate of this disease are high in Jinan city.Neonatal genetic screening is an important supplement to neonatal biochemical screening.Carrier screening for MMA-related pathogenic genes is recommended for couples of childbearing age in Jinan.

7.
International Journal of Traditional Chinese Medicine ; (6): 945-952, 2023.
Article in Chinese | WPRIM | ID: wpr-989732

ABSTRACT

Objective:To explore the effect of Xingnao Kaiqiao acupuncture treatment on brain network reorganization for the patients with stroke recovery, and therefore understand the neural mechanism underlying Xingnao Kaiqiao acupuncture treatment.Methods:Prospective case series study. Thirteen acute ischemia stroke patients were recruited from the Department of Neurology, Shanghai Minhang Hospital of Integrated Traditional Chinese and Western Medicine from Aug 2018 to Oct 2019. They were treated with Xingnao Kaiqiao acupuncture once a day for 10 consecutive days in addition to routine treatments, and received clinical assessments before treatment and 14 days after treatment onset. EEG signals were recorded during the first acupuncture treatment, from before inserting the needles (the baseline), during needle retention, to after removal of the needles. The brain network was constructed using phase locking index, and its clustering coefficient (CC), characteristic path length (PL) and small-worldness (S) were analyzed using one-way repeated ANOVA.Results:Compared with the baseline, the CC of delta-band network (sparsity=0.10: t=3.306, P=0.006; 0.12: t=2.909, P=0.013; 0.14: t=2.331, P=0.038) and the PL of delta-band (sparsity=0.12: t=3.236, P=0.007; 0.14: t=2.754, P=0.017, 0.18: t=2.878, P=0.014) and alpha-band (sparsity=0.10: t=2.432, P=0.032) networks were significantly decreased during the needle retention stage. Clinical assessments demonstrated a significant treatment efficacy of Xingnao Kaiqiao acupuncture, and its efficacy which was indicated by improved NIHSS score, was significantly correlated with the CC changes in the delta band network from baseline to needle retention. The correlation was strongest when the network sparsity was 0.12 ( r=0.78, P=0.002). Conclusion:Xingnao Kaiqiao acupuncture can regulate the brain network of stroke patients in real time, and this immediate regulation maybe associated with its treatment effect.

8.
International Journal of Surgery ; (12): 349-353, 2023.
Article in Chinese | WPRIM | ID: wpr-989460

ABSTRACT

Objective:To analyze the clinical characteristics of obese adolescents undergoing metabolic and bariatric surgery for the purpose to guide clinical decision-making and practice of metabolic and bariatric surgery in adolescents.Methods:The perioperative clinical profile of 123 adolescent patients under the age of 21 years who underwent metabolic and bariatric surgery in Beijing Friendship Hospital, Capital Medical University from 2018 to 2021 were extracted, and compared their clinical data with the overall data of 6807 patients in the 2021 GC-MBD Annual Report. Analyzed the clinical characteristics of adolescent weight loss metabolic surgery patients from multiple aspects such as preoperative complications, surgical methods, surgical outcomes, and follow-up. Statistical analysis was conducted using SPSS25.0 software.Results:Among adolescent bariatric surgery patients, the proportion of female patients (74.0%) was higher than that of male patients (26.0%). Except for the incidence of polycystic ovary syndrome (31.9%), which was higher than the overall rate, the proportion of other related diseases or symptoms was low and usually mild. The changes of systolic blood pressure ( Z=-5.73, P<0.001), body weight ( Z=-5.69, P<0.001), umbilical abdominal circumference ( Z=-2.40, P=0.017), glycosylated hemoglobin ( Z=-5.23, P<0.001), fasting insulin ( Z=-2.95, P<0.003), fasting C-peptide ( Z=-4.59, P<0.001), triglyceride ( Z=-2.75, P=0.006) at 3 months after operation were statistically significant compared with those before operation. The changes of systolic blood pressure ( Z=-3.42, P=0.001), body weight ( Z=-5.14, P<0.001), umbilical abdominal circumference ( Z=-2.86, P=0.004) and glycosylated hemoglobin ( Z=-2.67, P<0.008), fasting C-peptide ( Z=-2.09, P=0.037), high-density lipoprotein ( Z=-2.08, P=0.038) at 6 months after operation were statistically significant compared with those before operation. Conclusions:The outcomes of bariatric surgery in obese adolescents are similar to those in adults. The indications and timing of bariatric surgery need to be further explored. In view of the high proportion of females, poor self-management ability and low follow-up compliance of adolescent patients, it is urgent to construct a full-cycle care model suitable for adolescent patients with bariatric surgery to improve their self-management ability, follow-up compliance and long-term clinical outcomes.

9.
Chinese journal of integrative medicine ; (12): 825-831, 2023.
Article in English | WPRIM | ID: wpr-1010278

ABSTRACT

OBJECTIVE@#To investigate the anti-inflammatory activity of Radix Panacis quinguefolii root extract (RPQE) and its therapeutic effects on inflammatory bowel disease (IBD).@*METHODS@#The 72-hour post-fertilization zebrafish was used to generate the local and systematic inflammation models through tail-amputation and lipopolysaccharide (LPS)-induction (100 µ g/mL), respectively. The Tg(zlyz:EGFP) zebrafish was induced with 75 µ g/mL 2,4,6-trinitrobenzene sulfonic acid (TNBS) for establishing the IBD model. The tail-amputated, LPS-, and TNBS-induced models were subjected to RPQE (ethanol fraction, 10-20 µ g/mL) administration for 12 and 24 h, respectively. Anti-inflammatory activity of RPQE was evaluated by detecting migration and aggregation of leukocytes and expression of inflammation-related genes. Meanwhile, TNBS-induced fish were immersed in 0.2% (W/V) calcein for 1.5 h and RPQE for 12 h before photographing to analyze the intestinal efflux efficiency (IEE). Moreover, the expression of inflammation-related genes in these fish was detected by quantitative polymerase chain reaction.@*RESULTS@#Subject to RPQE administration, the migration and aggregation of leukocytes were significantly alleviated in 3 zebrafish models (P<0.01). Herein, RPQE ameliorated TNBS-induced IBD with respect to a significantly reduced number of leukocytes, improved IEE, and inhibited gene expression of pro-inflammatory factors (P<0.05 or P<0.01).@*CONCLUSION@#RPQE exhibited therapeutic effects on IBD by inhibiting inflammation.


Subject(s)
Animals , Zebrafish , Lipopolysaccharides , Disease Models, Animal , Inflammatory Bowel Diseases/metabolism , Inflammation/drug therapy , Anti-Inflammatory Agents/therapeutic use , Trinitrobenzenesulfonic Acid/adverse effects , Colitis/drug therapy
10.
China Journal of Chinese Materia Medica ; (24): 1916-1926, 2023.
Article in Chinese | WPRIM | ID: wpr-981411

ABSTRACT

The immunomodulatory effect of Saposhnikoviae Radix polysaccharide(SRP) was evaluated based on the zebrafish mo-del, and its mechanism was explored by transcriptome sequencing and real-time fluorescence-based quantitative PCR(RT-qPCR). The immune-compromised model was induced by navelbine in the immunofluorescence-labeled transgenic zebrafish Tg(lyz: DsRed), and the effect of SRP on the density and distribution of macrophages in zebrafish was evaluated. The effect of SRP on the numbers of macrophages and neutrophils in wild-type AB zebrafish was detected by neutral red and Sudan black B staining. The content of NO in zebrafish was detected by DAF-FM DA fluorescence probe. The content of IL-1β and IL-6 in zebrafish was detected by ELISA. The differentially expressed genes(DEGs) of zebrafish in the blank control group, the model group, and the SRP treatment group were analyzed by transcriptome sequencing. The immune regulation mechanism was analyzed by Gene Ontology(GO)and Kyoto Encyclopedia of Genes and Genomes(KEGG)enrichment, and the expression levels of key genes were verified by RT-qPCR. The results showed that SRP could significantly increase the density of immune cells in zebrafish, increase the number of macrophages and neutrophils, and reduce the content of NO, IL-1β, and IL-6 in immune-compromised zebrafish. The results of transcriptome sequencing analysis showed that SRP could affect the expression level of immune-related genes on Toll-like receptor pathway and herpes simplex infection pathway to affect the release of downstream cytokines and interferon, thereby completing the activation process of T cells and playing a role in regulating the immune activity of the body.


Subject(s)
Animals , Zebrafish/genetics , Interleukin-6/genetics , Gene Expression Profiling , Cytokines/genetics , Macrophages , Transcriptome
11.
Acta Pharmaceutica Sinica ; (12): 852-855, 2023.
Article in Chinese | WPRIM | ID: wpr-978754

ABSTRACT

Nanomedicine is one of the most promising fields in biomedicine. Inorganic nanomaterials stand out among many nanomaterials due to their excellent physicochemical properties, stable chemical properties and high biocompatibility. As an inorganic nanomaterial, bismuth-based nanomaterials have the advantages of adjustable band gap, low toxicity, easy functionalization, large X-ray attenuation coefficient, high photothermal conversion efficiency and long cycle half-life, so they have good promising application in cancer diagnosis and treatment. This review summarizes the recent research progress of bismuth-based nanomaterials in tumor diagnosis, treatment and biosafety, which provides a theoretical basis for the design and exploitation of a new generation of bismuth-based nanomedicine systems.

12.
Acta Pharmaceutica Sinica B ; (6): 3008-3026, 2023.
Article in English | WPRIM | ID: wpr-982902

ABSTRACT

Many efforts have been made to understand excitotoxicity and develop neuroprotectants for the therapy of ischemic stroke. The narrow treatment time window is still to be solved. Given that the ischemic core expanded over days, treatment with an extended time window is anticipated. Bestrophin 1 (BEST1) belongs to a bestrophin family of calcium-activated chloride channels. We revealed an increase in neuronal BEST1 expression and function within the peri-infarct from 8 to 48 h after ischemic stroke in mice. Interfering the protein expression or inhibiting the channel function of BEST1 by genetic manipulation displayed neuroprotective effects and improved motor functional deficits. Using electrophysiological recordings, we demonstrated that extrasynaptic glutamate release through BEST1 channel resulted in delayed excitotoxicity. Finally, we confirmed the therapeutic efficacy of pharmacological inhibition of BEST1 during 6-72 h post-ischemia in rodents. This delayed treatment prevented the expansion of infarct volume and the exacerbation of neurological functions. Our study identifies the glutamate-releasing BEST1 channel as a potential therapeutic target against ischemic stroke with a wide time window.

13.
Chinese Journal of Hepatology ; (12): 677-680, 2023.
Article in Chinese | WPRIM | ID: wpr-986192

ABSTRACT

A normal liver can develop cirrhosis through long-term and repeated stimulation from various etiologies. Histological manifestations like the collapse of hepatic lobular structure (including microvascular structure) and the formation of pseudolobules can lead to portal hypertension and even decompensated cirrhosis. More and more evidence suggests that effective etiological treatment can not only delay but also reverse the progression of cirrhosis. The mechanism of cirrhosis reversal mainly includes the degradation of extracellular matrix, hepatocyte regeneration, and hepatic lobular remodeling. The "gold standard" for the evaluation of cirrhosis reversal at present is still a liver biopsy. Therefore, the histopathological evaluation of cirrhosis reversal is very important for determining the disease's prognosis, efficacy, and mechanism of exploration.


Subject(s)
Humans , Liver Cirrhosis/pathology , Liver/pathology , Hypertension, Portal , Hepatocytes/pathology , Prognosis
14.
Chinese Journal of Hepatology ; (12): 385-388, 2023.
Article in Chinese | WPRIM | ID: wpr-986142

ABSTRACT

Chinese Society of Hepatology and Chinese Society of Infectious Diseases, Chinese Medical Association update the guidelines for the prevention and treatment of chronic hepatitis B (version 2022) in 2022. The latest guidelines recommend more extensive screening and more active antiviral treating for hepatitis B virus infection. This article interprets the essential updates in the guidelines to help deepen understanding and better guide the clinical practice.


Subject(s)
Humans , Hepatitis B, Chronic/drug therapy , Hepatitis B/drug therapy , Hepatitis B virus , Antiviral Agents/therapeutic use , Gastroenterology
15.
Journal of Zhejiang University. Medical sciences ; (6): 701-706, 2023.
Article in English | WPRIM | ID: wpr-1009945

ABSTRACT

OBJECTIVES@#To retrospectively analyze the variation and characteristics of phenylalanine hydroxylase (PAH) gene, and to observe the long-term treatment effect and follow-up of newborns with PAH deficiency.@*METHODS@#Clinical data, treatment and follow-up results of 198 patients with PAH deficiency diagnosed by newborn screening in Jinan from 1996 to 2021 were collected. The genetic analysis of 55 patients with PAH deficiency diagnosed by newborn screening in Jinan and 213 patients referred from the surrounding areas of Jinan were summarized. Gene variations were checked by a customized Panel gene detection method. Blood phenylalanine-concentration and physical development indicators including height and weight were regularly monitored. Intellectual development was assessed using a neuropsychological development scale for patients aged 0-6 years and academic performance, and brain injury in patients was assessed using brain magnetic resonance imaging.@*RESULTS@#c.728G>A, c.158G>A, c.721C>T, c.1068C>A, c.611A>G variations were common in PAH gene. The genotype of c.158G>A variation is compound heterozygous variation, with mainly a mild hyperpheny-lalaninemia. 168 patients with PAH deficiency who were followed-up regularly had normal physical development without dwarfism or malnutrition. Among the 33 preschool patients who underwent mental development assessment, 2 were mentally retarded and the initial treatment age was older than 6 months. Nine patients with an average age of (17.13±2.42) years completed brain magnetic resonance imaging, one case was normal, and 8 cases were abnormal. There were patchy or patchy hyperintense foci near the bilateral lateral ventricles on T2WI, and the intellectual development was normal. Compared with the other eight patients, the blood phenylalanine concentration of the normal child was better and stably controlled within the ideal range.@*CONCLUSIONS@#c.728G>A, c.158G>A, c.721C>T, c.1068C>A, c.611A>G variations were common in PAH gene. After standardized treatment, most patients with PAH deficiency diagnosed by screening can obtain normal growth and intellectual development in adolescence, but there are different degrees of organic lesions in the cerebral white matter.


Subject(s)
Child , Child, Preschool , Adolescent , Humans , Infant, Newborn , Young Adult , Adult , Neonatal Screening , Follow-Up Studies , Retrospective Studies , Phenylketonurias/genetics , Phenylalanine Hydroxylase/genetics , Phenylalanine/therapeutic use , Mutation
16.
Chinese Journal of Contemporary Pediatrics ; (12): 1143-1149, 2023.
Article in Chinese | WPRIM | ID: wpr-1009861

ABSTRACT

OBJECTIVES@#To investigate the changes in the serum levels of Klotho, fibroblast growth factor 23 (FGF23), and insulin-like growth factor-1 (IGF-1) in children with idiopathic short stature (ISS) before and after recombinant human growth hormone (rhGH) treatment, as well as the correlation of Klotho and FGF23 with the growth hormone (GH)/IGF-1 growth axis in these children.@*METHODS@#A prospective study was conducted on 33 children who were diagnosed with ISS in the Department of Pediatrics, Hebei Provincial People's Hospital, from March 10, 2021 to December 1, 2022 (ISS group). Twenty-nine healthy children, matched for age and sex, who attended the Department of Child Healthcare during the same period, were enrolled as the healthy control group. The children in the ISS group were treated with rhGH, and the serum levels of Klotho, FGF23, and IGF-1 were measured before treatment and after 3, 6, and 9 months of treatment. A correlation analysis was conducted on these indexes.@*RESULTS@#There were no significant differences in the serum levels of IGF-1, Klotho, and FGF23 between the ISS and healthy control groups (P>0.05). The serum levels of Klotho, FGF23, and IGF-1 increased significantly in the ISS group after 3, 6, and 9 months of rhGH treatment (P<0.05). In the ISS group, Klotho and FGF23 levels were positively correlated with the phosphate level before treatment (P<0.05). Before treatment and after 3, 6, and 9 months of rhGH treatment, the Klotho level was positively correlated with the IGF-1 level (P<0.05), the FGF23 level was positively correlated with the IGF-1 level (P<0.05), and the Klotho level was positively correlated with the FGF23 level (P<0.05), while Klotho and FGF23 levels were not correlated with the height standard deviation of point (P>0.05).@*CONCLUSIONS@#The rhGH treatment can upregulate the levels of Klotho, FGF23, and IGF-1 and realize the catch-up growth in children with ISS. Klotho and FGF23 may not directly promote the linear growth of children with ISS, but may have indirect effects through the pathways such as IGF-1 and phosphate metabolism. The consistent changes in Klotho, FGF23 and IGF-1 levels show that there is a synergistic relationship among them in regulating the linear growth of ISS children.


Subject(s)
Child , Humans , Human Growth Hormone/pharmacology , Insulin-Like Growth Factor I/pharmacology , Fibroblast Growth Factor-23 , Prospective Studies , Growth Disorders , Phosphates/pharmacology , Body Height
17.
Acta Pharmaceutica Sinica ; (12): 3185-3190, 2023.
Article in Chinese | WPRIM | ID: wpr-999100

ABSTRACT

Good medicine tastes bitter, but it is often difficult to swallow because the drug is bitter and astringent, so that the compliance of patients with medication is poor. However, the use of taste masking technology can better improve this situation. Appropriate and effective taste masking technology can improve the drug compliance of patients, especially children, it can also improve the curative effect and the clinical value of drugs. Herein, we summarize the latest research progress of taste masking technology, and summarize the traditional taste masking technology from the aspects of action mechanisms and application scopes. Finally, the novel and efficient taste masking technologies were presented.

18.
Acta Pharmaceutica Sinica ; (12): 3179-3184, 2023.
Article in Chinese | WPRIM | ID: wpr-999099

ABSTRACT

The taste of drugs has an important impact on the compliance of patients, but most of the active drug ingredients have an uncomfortable taste, especially traditional Chinese medicine. Through a variety of pharmaceutical excipients with taste masking properties combined with corresponding technologies can improve the taste of drugs and the characteristics of other dosage forms, so as to improve patient compliance. Here, we mainly summarize the auxiliary materials used for taste masking, explain the mechanism of taste masking from the point of view of excipients and introduces related uses, so as to provide reference for further research on taste masking of pediatric preparations.

19.
Acta Pharmaceutica Sinica ; (12): 64-75, 2022.
Article in Chinese | WPRIM | ID: wpr-913168

ABSTRACT

As a basic amino acid, histidine has a pKa close to the acidity of the tumor microenvironment, thus the charge and solubility of histidine are able to vary as the pH changes. Under a neutral environment, histidine is not charged and exhibits hydrophobic properties, while it can be protonated and becomes hydrophilic when exposed to mildly acidic pH, such as tumor microenvironment. Therefore, histidine is widely used in the design of drug delivery systems to target the mildly acidic pH of tumor microenvironment. This article reviews the recent progresses of histidine-based tumor-targeting drug delivery systems, and summarizes the principles on promoting internalization and tuning drug release by taking advantage of histidine. Finally, we point out the common issues on histidine application and illustrate its future prospects.

20.
Clinical Medicine of China ; (12): 554-558, 2022.
Article in Chinese | WPRIM | ID: wpr-956417

ABSTRACT

Objective:To summarize the genetic characteristics of a case of spinal muscular atrophy type 1c.Methods:The case data of a child with spinal muscular atrophy type 1c was retrospectively analyzed, and the genetic analysis and literature review were carried out.Results:The patient, male, started at the age of 2 months, and showed gross motor development backwardness and low muscular tension. Multiplex connection probe amplification technique showed that the child had homozygous deletion mutation in exon 7-8 of SMN1 gene, and there was duplicate mutation in exon 7-8 of SMN2 gene. The number of copies of exon 7/8 was 3/3. His father was a heterozygous deletion carrier of SMN1 gene, and there was homozygous mutation in exon 8 of SMN2 gene. The number of copies of exon 7/8 was 2/3. His mother did not find abnormal exons of SMN1 gene, and the number of copies of exon 7/8 of SMN2 gene was 1/1.Conclusion:Spinal muscular atrophy lacks specific manifestations in the early stage, and the diagnosis mainly depends on genetic testing. Clinicians need to be vigilant, strengthen the early understanding of the disease, and improve the prognosis.

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